| 摘要: |
| 目的:了解美国食品药品监督管理局(FDA)儿童药物临床结局评估(COA)项目的现状,为我国儿童中药新药的研发提
供参考。方法:梳理美国FDA 已登记的儿童药物COA 项目类别、最新状态等,并结合申报资料及美国FDA 反馈意见进行分析。
结果:目前涉及儿童人群的药物COA 项目共18 个,有15 个按现行程序提交了具体内容。按现行程序提交的项目中,最新状态
为接受纳入资质认证计划的11 项,在适应证分类上以皮肤类最多,COA 类型上涉及患者报告结局的最多。结论:美国FDA 儿
童COA 工具现状具有重视尽早引入儿童药物的COA 项目研究、鼓励多类型COA 项目研发的特点。针对儿童中药COA 项目研
发存在的问题,可借鉴美国FDA 监管经验,加强多学科合作,共同推动儿童COA 项目研发。 |
| 关键词: 以患者为中心 药物研发 临床结局评估项目 儿童 中药 |
| DOI：doi:10.13407/j.cnki.jpp.1672-108X.2026.11.001 |
|
| 基金项目:基金项目:国家科技重大专项重大新药创制项目,编号2020ZX09201008。 |
|
| Current Status of the U. S. Food and Drug Administration Pediatric Clinical Outcome Assessment Projectand Its Implications for Patient-Focused Pediatric Traditional Chinese Medicine Research and Development |
| Jia Zhichao, Hu Siyuan, Xu Yaqian |
| (First Teaching Hospital of Tianjin University of Traditional Chinese Medicine, National
Clinical Research Center for Chinese Medicine, Tianjin 300381, China) |
| Abstract: |
| Objective: To probe into the current status of pediatric Clinical Outcome Assessment (COA) project developed by the U. S.
Food and Drug Administration (FDA), and to provide reference for the research and development of new pediatric traditional Chinese
medicine in China. Methods: The categories, latest status, and other relevant information of registered pediatric COA project were
retrieved from the U. S. FDA official website. Analysis was performed based on submission documents and FDA feedback. Results: A
total of 18 COA projects involving pediatric populations were identified, among which 15 had detailed content under current regulatory
procedures. Among the projects submitted according to the current procedure, 11 projects had been accepted into the qualification
program. By therapeutic areas, dermatology-related tools were the most prevalent. By COA type, patient-reported outcomes (PRO)
were the most frequently used. Conclusion: The current pediatric COA projects developed by the U. S. FDA highlights the importance
of early integration of COA studies into pediatric drug development, coverage of both rare and common diseases, and encouragement of
diverse COA project types. Given the challenges in developing COA projects for pediatric traditional Chinese medicine, it is advisable
to draw on FDA regulatory experience and strengthen multidisciplinary collaboration to jointly advance the development of pediatric
COA projects. |
| Key words: patient-focused drug research and development clinical outcome assessment children traditional Chinese medicine |
